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3.
At the end of the presentation, the attendee will be able to describe the genetics of Alport syndrome and the
impact of different mutations on the rapidity of progression to end-stage renal disease and deafness.
Presentation 3: Outcomes and Transplants, Patrick Niaudet, MD
Dr. Niaudet is a world-renowned pediatric nephrologist, who will discuss aspects of the clinical progression of
Alport disease manifestations and outcomes after renal transplantation, including current knowledge regarding
post-transplant anti-glomerular basement nephritis in the renal allograft.
At the end of the presentation, the attendee will be able to describe the typical clinical course of Alport kidney,
cochlear and ocular disease in affected males and females and the outcomes of renal transplantation.
Presentation 4: Pharmaceutical Treatment, Christoph Licht, MD
Dr. Licht will present published and unpublished data regarding the impact of pharmacologic therapies on Alport
kidney disease in experimental animals and in human populations.
At the end of the presentation, the attendee will be able to identify pharmacologic approaches that have shown
promise in experimental animals with Alport syndrome, and will understand the challenges involved in applying
these observations to people with Alport syndrome.
Presentation 5: Biological Treatment, Oliver Gross, PD, MD, FASN
Dr. Gross, who is Executive Director of the European Alport Registry, has performed numerous studies of Alport
syndrome therapies in experimental animals, including stem cell therapies. He will discuss the current status of
stem cell and gene replacement therapies for the disease.
At the end of the presentation, the attendee will be able to describe the findings of studies of stem cell therapies
in Alport syndrome, and the challenges associated with developing stem cell and gene replacement approaches
for treatment of human Alport syndrome.
Presentation 6: Hearing Loss, Dominic Cosgrove, PhD
Dr. Cosgrove will provide a state-of-the-art presentation on the role of basement membranes in cochlear
function and current theories regarding the pathogenesis of hearing loss in Alport syndrome.
At the end of the presentation, the attendee will be able to describe the alterations of cochlear architecture and
function found in Alport subjects, and how these changes may be related to hearing loss.
Presentation 7: Women and Alport Syndrome, Michelle Rheault, MD
Dr. Rheault has characterized the first transgenic model of X-linked Alport syndrome. She will describe new
findings regarding the role of X-chromosome inactivation in determining the outcome of X-linked Alport
syndrome in heterozygous females, and discuss how kidney donation by heterozygous females should be
approached.
At the end of the presentation, the attendee will be able to describe how X-chromosome inactivation influences
the course and outcome of X-linked Alport syndrome in heterozygous females.
Presentation 8: Registries/Trials – Alport Syndrome Treatments and Outcomes Registry (ASTOR),
Clifford E. Kashtan, MD
Dr. Kashtan is the Executive Director of the Alport Syndrome Treatments and Outcomes Registry (ASTOR). He
will discuss the roles of registries in the United States and Europe in supporting clinical studies of Alport
syndrome therapies and provide an update on new findings of registry studies. He will also present an overview
of the clinical research process.
At the end of the presentation, the attendee will be able to discuss the latest findings of studies carried out by
Alport syndrome registries.
Presentation 9: Research Process Overview, Clifford E. Kashtan, MD
Dr. Kashtan will present an overview of the clinical research process including a description of the activities
associated with design, development and implementation of clinical research policies and procedures.

4.
At the end of the presentation, the attendee will understand the complexities of designing and implementing
clinical research studies of rare genetic disorders.
Presentation 10: Children and Adolescents with Chronic Kidney Disease, Bradley Warady, MD
Dr. Warady is one of the world's authorities on chronic renal failure and end-stage renal disease in children and
adolescents, and on the challenges of insuring adherence with dietary prescriptions and medication regimens in
adolescent patients. He will present current information on the causes and correction of dietary and medication
non-adherence.
At the end of the presentation, the attendee will be able to discuss the causes and approaches to correction of
dietary and medication non-adherence in adolescent patients.